tbuckley/cabot-search huggingface dataset:
Cost-effectiveness and Return on Investment of a Nationwide Case-Finding Program for Familial Hypercholesterolemia in Children in the Netherlands
Authors: Zanfina Ademi, Richard Norman, Jing Pang, Eric Sijbrands, Gerald F. Watts, Barbara A. Hutten, Albert Wiegman
Journal: JAMA Pediatrics (2023)

Importance The Netherlands is one of the few countries that has a long-term history of active screening for familial hypercholesterolemia (FH), enabling health-economic analyses. Objective To investigate cost-effectiveness and the return on investment (ROI) of a nationwide cascade case-finding and preventive treatment program starting with identification of FH in children and treatment, from both a societal and health care perspective. Design, Setting, and Participants Cascade case-finding and early preventive treatment were modeled to simulate the progression of disease and costs of 10-year-olds suspected of having heterozygous FH over a lifetime. The model consisted of 3 health states: alive without coronary heart disease (CHD), alive with CHD, and deceased. Mendelian randomization analysis was used to quantify the risk of a first CHD event as a function of age and total lifetime exposure to low-density lipoprotein cholesterol. Cost-effectiveness was defined as €20 000 ($21 800) per QALYs (quality-adjusted life-years) gained, using incremental cost-effectiveness ratios (ICERs). All future benefits and costs were discounted annually by 1.5% and 4%, respectively. Interventions The study compared 2 strategies: (1) cascade screening and initiation of treatment with statins in children (mean age, 10 years) and (2) no screening, later detection, and treatment. Main Outcomes and Measures Outcome of interest included cost, detection, and successful treatment of FH in terms of life-years gained and QALYs. The clinical and cost outputs for each model in the 2 scenarios (early detection and treatment and later detection and treatment) were totaled to determine the overall cost-effectiveness and ROI attributed to implementation of the Dutch FH program. Results In this model constructed to simulate the progression of FH in 1000 hypothetical 10-year-olds, from a health care perspective, the program would gain 2.53 QALYs per person, at an additional cost of €23 365 ($25 468) (both discounted). These equated to an ICER of €9220 ($10 050) per QALY gained. From the societal perspective, the detection and treatment program were cost saving over a lifetime compared with no cascade screening for FH. The ROI for the detection and treatment program for FH in children was €8.37 ($9.12). Conclusions and Relevance The findings of this study suggest that the early detection and treatment program for FH in children may offer a good value for investment, being both health and cost saving. The findings and interpretations are conditional on assumptions inherent in the health economic model.
The Disappointing Impact of Interventions to Prevent Hospital Readmissions
Authors: Robert M. Wachter
Journal: JAMA Internal Medicine (2023)

Our website uses cookies to enhance your experience. By continuing to use our site, or clicking "Continue," you are agreeing to our Cookie Policy | Continue JAMA Internal Medicine HomeNew OnlineCurrent IssueFor Authors Podcast Journals JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry (1919-1959) JN Learning / CMESubscribeJobsInstitutions / LibrariansReprints & Permissions Terms of Use | Privacy Policy | Accessibility Statement 2024 American Medical Association. All Rights Reserved Search All JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Forum Archive JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry Input Search Term Sign In Individual Sign In Sign inCreate an Account Access through your institution Sign In Purchase Options: Buy this article Rent this article Subscribe to the JAMA Internal Medicine journal
Xuebijing Injection for the Treatment of Sepsis
Authors: Ellis F. Unger, David B. Clissold
Journal: JAMA Internal Medicine (2023)

Our website uses cookies to enhance your experience. By continuing to use our site, or clicking "Continue," you are agreeing to our Cookie Policy | Continue JAMA Internal Medicine HomeNew OnlineCurrent IssueFor Authors Podcast Journals JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry (1919-1959) JN Learning / CMESubscribeJobsInstitutions / LibrariansReprints & Permissions Terms of Use | Privacy Policy | Accessibility Statement 2024 American Medical Association. All Rights Reserved Search All JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Forum Archive JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry Input Search Term Sign In Individual Sign In Sign inCreate an Account Access through your institution Sign In Purchase Options: Buy this article Rent this article Subscribe to the JAMA Internal Medicine journal
Escinosome thermosensitive gel optimizes efficacy of CAI-CORM in a rat model of rheumatoid arthritis
Authors: Giulia Vanti, Laura Micheli, Emanuela Berrino, Lorenzo Di Cesare Mannelli, Irene Bogani, Fabrizio Carta, Maria Camilla Bergonzi, Claudiu T. Supuran, Carla Ghelardini, Anna Rita Bilia
Journal: Journal of Controlled Release (2023)

Rheumatoid arthritis is among the most common disabling diseases associated with chronic inflammation. The efficacy of the current therapeutic strategies is limited; therefore, new pharmacological agents and formulation approaches are urgently needed. In this work, we developed a thermosensitive gel incorporating escinosomes, innovative nanovesicles made of escin, stabilized with 10% of tween 20 and loaded with a Carbonic Anhydrase Inhibitor (CAI) bearing a Carbon Monoxide Releasing Moiety (CORM) (i.e., CAI-CORM 1), previously synthesized by some of the authors as a new potent pain-relieving agent. The light scattering analysis of the developed formulation showed optimal physical parameters, while the chromatographic analysis allowed the quantification of the encapsulation efficiency (90.1 ± 5.91 and 91.6 ± 8.46 for CAI-CORM 1 and escin, respectively). The thermosensitive gel, formulated using 23% w/v of poloxamer 407, had a sol-gel transition time of 40 s and good syringeability. Its stability in simulated synovial fluid (SSF) was morphologically evaluated by electron microscopy. Nanovesicles were physically stable in contact with the medium for two weeks, maintaining their original dimensions and spherical shape. The viscosity increased by about 30- to 100-fold with the temperature change from 25 °C to 37 °C. The gel erosion in SSF occurred within 9 h (88.2 ± 0.743%), and the drug's passive diffusion from escinosomes lasted 72 h, allowing a potential sustained therapeutic effect. The efficacy of a single intra-articular injection of the gel containing escinosomes loaded with CAI-CORM 1 (3 mg/mL; 30 μL, CAI-CORM 1 formulation) and the gel containing unloaded escinosomes (30 μL, blank formulation) was evaluated in a rat model of Complete Freund's Adjuvant (CFA)–induced rheumatoid arthritis. CAI-CORM 1 formulation was assessed to counteract mechanical hyperalgesia, spontaneous pain, and motor impairments on days 7 and 14 after treatment. The histological evaluation of the joints stressed the improvement of several morphological parameters in CFA + CAI-CORM 1 formulation-treated rats. In conclusion, the hybrid molecule CAI-CORM 1 formulated in escinosome-based thermosensitive gel could represent a new valid approach for managing rheumatoid arthritis.
Designing Health Care Artificial Intelligence That Comports With the Values of Patients—Children Are People Whose Voices Must Be Heard
Authors: Debra J. H. Mathews, Kadija Ferryman, Ruth Faden
Journal: JAMA Network Open (2023)

Kelly Thai, MPH; Kate H. Tsiandoulas, BA; Elizabeth A. Stephenson, MD; Dolly Menna-Dack, MHSc; Randi Zlotnik Shaul, PhD; James A. Anderson, PhD; Alexis R. Shinewald, CCLS; Augustina Ampofo, BA; Melissa D. McCradden, PhD
What Is the Optimum Approach to Screening for Familial Hypercholesterolemia in Children?
Authors: Stephen R. Daniels
Journal: JAMA Pediatrics (2023)

Our website uses cookies to enhance your experience. By continuing to use our site, or clicking "Continue," you are agreeing to our Cookie Policy | Continue JAMA Pediatrics HomeNew OnlineCurrent IssueFor Authors Podcast Journals JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry (1919-1959) JN Learning / CMESubscribeJobsInstitutions / LibrariansReprints & Permissions Terms of Use | Privacy Policy | Accessibility Statement 2023 American Medical Association. All Rights Reserved Search All JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Forum Archive JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry Input Search Term Sign In Individual Sign In Sign inCreate an Account Access through your institution Sign In Purchase Options: Buy this article Rent this article Subscribe to the JAMA Pediatrics journal
Efficient colonic drug delivery in domestic pigs employing a tablet formulation with dual control concept
Authors: Viviane Doggwiler, Chasper Puorger, Valeria Paredes, Michael Lanz, Katja M. Nuss, Georg Lipps, Georgios Imanidis
Journal: Journal of Controlled Release (2023)

Efficient and reproducible colonic drug delivery remains elusive. The aim of this study was to demonstrate specific colonic delivery in vivo in domestic pigs with a novel tablet formulation based on a dual release control concept using 5-aminosalicylic acid (5-ASA) and caffeine as drug substances. The developed controlled colonic release (CCR) tablet formulation employs a pH-sensitive coating based on Eudragit® FS 30 D to prevent drug release in the upper gastrointestinal tract, and a xyloglucan-based matrix to inhibit drug release after coating removal in the small intestine and to allow microbiome-triggered drug release by enzymatic action in the colon. CCR tablets were administered to domestic pigs and plasma concentration data was analyzed by physiologically based pharmacokinetic modeling to estimate absorbed amounts from small and large intestine and in vivo drug release rates by model-dependent deconvolution using immediate release (IR) tablets and intravenous solutions as reference. Peak concentration times (tmax) and values (cmax) of CCR 5-ASA and caffeine tablets indicated strongly delayed drug absorption and the deduced absorbed amount as a function of time confirmed absorption overwhelmingly from the large intestine. The microbially cleaved marker molecule sulfasalazine administered alone or together with caffeine in CCR tablets reported, in combination with telemetry measurements, gastrointestinal transit times and site of absorption. Drug release from CCR tablets was inferred to take place predominantly at the site of absorption at a release rate of caffeine that was much larger in the colon than in the small intestine indicating enzymatically triggered release by the colonic microbiome. Xyloglucanase activity in rectal and cecal samples was consistent with release data and compound recovery in fecal droppings was consistent with 5-ASA bioavailability. The results provide evidence that the developed formulation can prevent premature drug release and provide targeted colonic drug delivery. Clinical relevance based on the comparability between pig and man is discussed.
Identification of a Common Variant for Coronary Heart Disease at <i>PDE1A</i> Contributes to Individualized Treatment Goals and Risk Stratification of Cardiovascular Complications in Chinese Patients With Type 2 Diabetes
Authors: Claudia H.T. Tam, Cadmon K.P. Lim, Andrea O.Y. Luk, Mai Shi, Hoi Man Cheung, Alex C.W. Ng, Heung-man Lee, Eric S.H. Lau, Baoqi Fan, Guozhi Jiang, Alice P.S. Kong, Risa Ozaki, Elaine Y.K. Chow, Ka Fai Lee, Shing Chung Siu, Grace Hui, Chiu Chi Tsang, Kam Piu Lau, Jenny Y.Y. Leung, Elaine Y.N. Cheung, Man Wo Tsang, Grace Kam, Ip Tim Lau, June K.Y. Li, Vincent T.F. Yeung, Emmy Lau, Stanley Lo, Samuel Fung, Yuk Lun Cheng, Chun Chung Chow, Xiaodan Fan, Ting Fung Chan, Kevin Y.L. Yip, Si Lok, Weichuan Yu, Stephen K.W. Tsui, Hui-yao Lan, Cheuk Chun Szeto, Nelson L.S. Tang, Brian Tomlinson, Yu Huang, Alicia J. Jenkins, Anthony Keech, Wing-yee So, Juliana C.N. Chan, Ronald C.W. Ma, Ronald C.W. Ma, Juliana C.N. Chan, Risa Ozaki, Andrea O.Y. Luk, Wingyee So, Ka-fai Lee, Shing-chung Siu, Grace Hui, Chiu-chi Tsang, Kam-piu Lau, Jenny Y.Y. Leung, Man-wo Tsang, Grace Kam, Elaine Cheung, Ip-tim Lau, June K.Y. Li, Vincent T.F. Yeung, Jo Jo Kwan, Samuel Fung, Stanley Lo, Emmy Lau, Yuk-lun Cheng, Stephen K.W. Tsui, Yu Huang, Huiyao Lan, Weichuan Yu, Brian Tomlinson, Si Lok, Ting-fung Chan, Kevin Y.L. Yip, Cheuk-chun Szeto, Xiaodan Fan, Nelson L.S. Tang, Xiaoyu Tian, Claudia H.T. Tam, Guozhi Jiang, Shi Mai, Baoqi Fan, Fei Xie, Sen Zhang, Pu Yu, Meng Wang, Heung-man Lee, Cadmon K.P. Lim, Fangying Xie, Alex C.W. Ng, Grace P.Y. Cheung, Alice P.S. Kong, Elaine Y.K. Chow, Ming-wai Yeung, Chun-chung Chow, Kitty K.T. Cheung, Rebecca Y.M. Wong, Honcheong So, Katie K.H. Chan, Chin-san Law, Anthea K.Y. Lock, Ingrid K.Y. Tsang, Susanna C.P. Chan, Yin-wah Chan, Cherry Chiu, Chi-sang Hung, Cheuk-wah Ho, Ivy H.Y. Ng, Maria W.H. Mak, Kai-man Lee, Candy H.S. Leung, Ka-wah Lee, Hui-ming Chan, Winnie Wat, Tracy Lau, Cheuk-yiu Law, Ryan H.Y. Chan, Candice Lau, Pearl Tsang, Vince Chan, Lap-ying Ho, Eva Wong, Josephine Chan, Sau-fung Lam, Jessy Pang, Yee-mui Lee, Ronald C.W. Ma, Juliana C.N. Chan, Yu Huang, Hui-yao Lan, Si Lok, Brian Tomlinson, Stephen K.W. Tsui, Weichuan Yu, Kevin Y.L. Yip, Ting-fung Chan, Xiaodan Fan, Wing-yee So, Cheuk-chun Szeto, Nelson L.S. Tang, Andrea O.Y. Luk, Xiaoyu Tian, Claudia H.T. Tam, Guozhi Jiang, Heung Man Lee, Cadmon K.P. Lim, Katie K.H. Chan, Fangying Xie, Alex C.W. Ng, Grace P.Y. Cheung, Ming-wai Yeung, Shi Mai, Fei Xie, Wei Jiang, Sen Zhang, Pu Yu, Meng Weng, Kelly Y. Li, Chuiguo Huang, Gechang Yu
Journal: Diabetes Care (2023)

In this study we aim to unravel genetic determinants of coronary heart disease (CHD) in type 2 diabetes (T2D) and explore their applications.
Preexisting Financial Hardship Among Caregivers of Hospitalized Children
Authors: Erin F. Carlton, Michelle H. Moniz, John W. Scott, Hallie C. Prescott, Lisa A. Prosser, Nora V. Becker
Journal: JAMA Pediatrics (2023)

This cross-sectional study examines preexisting financial hardship among caregivers of hospitalized children.
Changes in Depression and Anxiety Among Children and Adolescents From Before to During the COVID-19 Pandemic
Authors: Sheri Madigan, Nicole Racine, Tracy Vaillancourt, Daphne J. Korczak, Jackson M. A. Hewitt, Paolo Pador, Joanne L. Park, Brae Anne McArthur, Celeste Holy, Ross D. Neville
Journal: JAMA Pediatrics (2023)

There is a growing body of high-quality cohort-based research that has examined changes in child and adolescent mental health during the COVID-19 pandemic vs before the pandemic. Some studies have found that child and adolescent depression and anxiety symptoms have increased, while others have found these symptoms to have remained stable or decreased.
Patient Experience at US Hospitals Following the Caregiver Advise, Record, Enable (CARE) Act
Authors: Courtney R. Lee, Elizabeth Taggert, Norma B. Coe, Paula Chatterjee
Journal: JAMA Network Open (2023)

Importance Communication with caregivers is often not established or standardized during hospitalization. The Caregiver Advise, Record, Enable (CARE) Act is a state-level policy designed to facilitate communication among patients, caregivers, and clinical care teams during hospitalization to improve patient experience; 42 states have passed this policy since 2014, but whether it was associated with achieving these goals remains unknown. Objective To determine whether passage of the CARE Act was associated with improvements in patient experience. Design, Setting, and Participants This cohort study used a difference-in-differences analysis of short-term, acute-care US hospitals from 2013 to 2019 to analyze changes in patient experience before vs after CARE Act implementation in hospitals located in states that passed the CARE Act compared with those in states that did not. Analyses were performed between September 1, 2021, and July 31, 2022. Exposure Time-varying indicators for whether a hospital was in a state that passed the CARE Act. Main Outcomes and Measures Patient-reported experience via the Hospital Consumer Assessment of Healthcare Providers and Systems survey. Results A total of 2763 hospitals were included, with 2188 hospitals in CARE Act states and 575 in non–CARE Act states. There were differential improvements in patient experience in the measures of communication with nurses (unadjusted mean [SD] score, 78.40% [0.42%]; difference, 0.18 percentage points; 95% CI, 0.07-0.29 percentage points; P = .002), communication with physicians (mean [SD] score, 80.00% [0.19%]; difference, 0.17 percentage points; 95% CI, 0.06-0.28 percentage points; P = .002), and receipt of discharge information (mean [SD] score, 86.40% [0.22%]; difference, 0.11 percentage points; 95% CI, 0.02-0.21 percentage points; P = .02) among CARE Act states compared with non–CARE Act states after policy passage. In subgroup analyses, improvements were larger among hospitals with lower baseline Hospital Consumer Assessment of Healthcare Providers and Systems performance on measures of communication with nurses, communication with physicians, and overall hospital rating. Conclusions and Relevance These findings suggest that implementation of the CARE Act was associated with improvements in several measures of patient experience. Policies that formally incorporate caregivers into patient care during hospitalization may improve patient outcomes.
Direct-to-Patient Clinical Trials: One Small Step or a Giant Leap?
Authors: Aakash Desai, Vivek Subbiah
Journal: Clinical Cancer Research (2023)

Genome driven precision oncology has transformed the landscape of multiple cancers. However, access barriers exist. A recent study exemplified a direct-to-patient outreach program via social media through the implementation of a global program that offered free tumor genomic testing with a focus on rare cancers. See related article by Doe-Tetteh et al., p. 2445.
Cystic Fibrosis–Related Diabetes Workshop: Research Priorities Spanning Disease Pathophysiology, Diagnosis, and Outcomes
Authors: Melissa S. Putman, Andrew W. Norris, Rebecca L. Hull, Michael R. Rickels, Lori Sussel, Scott M. Blackman, Christine L. Chan, Katie Larson Ode, Tanicia Daley, Arlene A. Stecenko, Antoinette Moran, Meagan J. Helmick, Sharon Cray, Jessica A. Alvarez, Virginia A. Stallings, Katherine L. Tuggle, John P. Clancy, Thomas L. Eggerman, John F. Engelhardt, Andrea Kelly
Journal: Diabetes Care (2023)

Cystic fibrosis (CF) is a recessive disorder arising from mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CFTR is expressed in numerous tissues, with high expression in the airways, small and large intestine, pancreatic and hepatobiliary ducts, and male reproductive tract. CFTR loss in these tissues disrupts regulation of salt, bicarbonate, and water balance across their epithelia, resulting in a systemic disorder with progressive organ dysfunction and damage. Pancreatic exocrine damage ultimately manifests as pancreatic exocrine insufficiency that begins as early as infancy. Pancreatic remodeling accompanies this early damage, during which abnormal glucose tolerance can be observed in toddlers. With increasing age, however, insulin secretion defects progress such that CF-related diabetes (CFRD) occurs in 20% of teens and up to half of adults with CF. The relevance of CFRD is highlighted by its association with increased morbidity, mortality, and patient burden. While clinical research on CFRD has greatly assisted in the care of individuals with CFRD, key knowledge gaps on CFRD pathogenesis remain. Furthermore, the wide use of CFTR modulators to restore CFTR activity is changing the CFRD clinical landscape and the field’s understanding of CFRD pathogenesis. For these reasons, the National Institute of Diabetes and Digestive and Kidney Diseases and the Cystic Fibrosis Foundation sponsored a CFRD Scientific Workshop, 23–25 June 2021, to define knowledge gaps and needed research areas. This article describes the findings from this workshop and plots a path for CFRD research that is needed over the next decade.
Effects of a Multimodal Transitional Care Intervention in Patients at High Risk of Readmission
Authors: Jacques Donzé, Gregor John, Daniel Genné, Marco Mancinetti, Alexandre Gouveia, Marie Méan, Lukas Bütikofer, Drahomir Aujesky, Jeffrey Schnipper
Journal: JAMA Internal Medicine (2023)

Hospital readmissions are frequent, costly, and sometimes preventable. Although these issues have been well publicized and incentives to reduce them introduced, the best interventions for reducing readmissions remain unclear.To evaluate the effects of a multimodal transitional care intervention targeting patients at high risk of hospital readmission on the composite outcome of 30-day unplanned readmission or death.A single-blinded, multicenter randomized clinical trial was conducted from April 2018 to January 2020, with a 30-day follow-up in 4 medium-to-large-sized teaching hospitals in Switzerland. Participants were consecutive patients discharged from general internal medicine wards and at higher risk of unplanned readmission based on their simplified HOSPITAL score (≥4 points). Data were analyzed between April and September 2022.The intervention group underwent systematic medication reconciliation, a 15-minute patient education session with teach-back, a planned first follow-up visit with their primary care physician, and postdischarge follow-up telephone calls from the study team at 3 and 14 days. The control group received usual care from their hospitalist, plus a 1-page standard study information sheet.Thirty-day postdischarge unplanned readmission or death.A total of 1386 patients were included with a mean (SD) age of 72 (14) years; 712 (51%) were male. The composite outcome of 30-day unplanned readmission or death was 21% (95% CI, 18% to 24%) in the intervention group and 19% (95% CI, 17% to 22%) in the control group. The intention-to-treat analysis risk difference was 1.7% (95% CI, -2.5% to 5.9%; P = .44). There was no evidence of any intervention effects on time to unplanned readmission or death, postdischarge health care use, patient satisfaction with the quality of their care transition, or readmission costs.In this randomized clinical trial, use of a standardized multimodal care transition intervention targeting higher-risk patients did not significantly decrease the risks of 30-day postdischarge unplanned readmission or death; it demonstrated the difficulties in preventing hospital readmissions, even when multimodal interventions specifically target higher-risk patients.ClinicalTrials.gov Identifier: NCT03496896.